Bifunctional Biologic Gets Orphan Drug Status for Paroxysmal Nocturnal Hemoglobinuria
KP104 is a bifunctional, first-in-class biologic that works by selectively inhibiting the alternative and terminal pathways in the complement system.
KP104 is a bifunctional, first-in-class biologic that works by selectively inhibiting the alternative and terminal pathways in the complement system.
Beti-cel is an investigational one-time gene therapy that works by adding functional copies of a modified form of the beta-globin gene into a patient’s own hematopoietic stem cells.
The application included data from the phase 2 BEYOND study.
The NDA is supported by data from 5 ASCEND phase 3 clinical trials.
BCX9930 is an investigational oral Factor D inhibitor.
In a study of incident dialysis patients, the HIF-PHI agent daprodustat demonstrated safety and efficacy similar to that of the ESA darbepoetin alfa.
By clarifying the risk for anaphylaxis, study findings can contribute to the choice of IV iron preparations, according to investigators.
A review of regulatory and drug development status updates.
The NDA submission included data from 36 clinical trials involving over 8000 patients.
The FDA has approved Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.